Enzyme Replacement Therapy Market Analysis by Product (Agalsidase Beta, Imiglucerase, Velaglucerase Alfa, Idursulfase, Galsulfase, and Laronidase); by Disease (Gaucher Disease, Fabry Disease, Pompe Disease, Mucopolysaccharidosis, and Exocrine Pancreatic Insufficiency [EPI]); and End User (Hospitals, and Infusion Centers & Home Healthcare Settings) – Global Supply & Demand Analysis & Opportunity Outlook 2023-2033

Buy Now Report ID: 4455 | Published Date: Feb 27, 2023

Global Enzyme Replacement Therapy Market Size, Forecast, and Trend Highlights Over 2023 - 2033

The global enzyme replacement therapy market is estimated to garner a revenue of ~ USD 14 Billion by the end of 2033 by growing at a CAGR of ~7% over the forecast period, i.e., 2023 – 2033. Further, the market generated a revenue of ~ USD 10 Billion in the year 2022. The growth of the market can be attributed to rising prevalence of lysosomal storage diseases (LSDs) in the population owing to rising diseases and an unhealthy lifestyle. There were 766 LSD diagnoses in the Australian population during the 12-year period, January 2009 through December 2020, including 32 successful pregnancies.

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Also, the growing population is expected to boost the market further over the forecast period. The growth in population is anticipated to lead a rise in the prevalence of rare diseases, which is also expected to fuel the demand for enzyme replacement therapy. The population of the world is projected to rise by 2 billion people during the next 30 years, from 7.7 billion people today to 9.7 billion in 2050, with a potential peak of approximately 11 billion people around the year 2100.

The worldwide incidence of lysosomal storage illnesses, including Gaucher, Fabry, Pompe, and MPS, is steadily rising. For instance, as per information released by the National Institute of Neurological Disorders and Stroke in August 2021, approximately one in 40,000 individuals in the United States had Pompe disease, which is predicted to be roughly 32,950 individuals. Furthermore, the National Fabry Foundation estimates that the entire Fabry patient number in the USA will be roughly 7,713 in May 2020. As a result of the significant number of patients experiencing rare lysosomal storage illnesses, there is a huge requirement for efficient treatments, including enzyme replacement therapy.

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Global Enzyme Replacement Therapy Market: Key Insights

Base Year


Forecast Year




Base Year Market Size (2022)

~ USD 10 Billion

Forecast Year Market Size (2033)

~ USD 14 Billion

Regional Scope

  • North America (U.S., and Canada)
  • Latin America (Mexico, Argentina, Rest of Latin America)
  • Asia-Pacific (Japan, China, India, Indonesia, Malaysia, Australia, Rest of Asia-Pacific)
  • Europe (U.K., Germany, France, Italy, Spain, Russia, NORDIC, Rest of Europe)
  • Middle East and Africa (Israel, GCC North Africa, South Africa, Rest of Middle East and Africa)

Global Enzyme Replacement Therapy Market: Growth Drivers and Challenges


Growth Drivers

  • Growing Government Spending on Healthcare Infrastructure – Governments of different nations are focusing on expanding the healthcare sector to treat people and combating health issues. As a result of rising investment in healthcare infrastructure, the market size of enzyme replacement therapy is expected to expand in the assessment period. According to an estimate, in 2020, the United States spent more than USD 3 trillion on healthcare.

  • Rise in Prevalence of Rare Disease - There are between 1,000 and 2,000 new occurrences of ALSP each year, which is a rare, inherited, autosomal dominant neurological condition with strong penetrance that affects an estimated 10,000 persons in the US.

  • Growing Cases of Pompe Disease - Since enzyme replacement therapy (ERT) serves as the only viable treatment option for Pompe disease. Therefore, increasing cases of the illness are anticipated to contribute to market expansion. ERT has contributed to disease course stabilization with motor and pulmonary modifications in patient populations with late-onset Pompe disease. According to experts, there are between approximate 4,000 and about 9,000 cases of Pompe disease globally.

  • Technology Advancement - The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended that Lamzede (velmanase alfa), a long-term enzyme replacement therapy for adults, adolescents, and children with mild to moderate forms of alpha-mannosidosis, be given marketing authorization in the European Union (EU).

  • Growing Genetic Disorder - A genetically based ailment will affect roughly 6 out of every 10 individuals across the globe.


  • Lack of Awareness - Lack of awareness about enzyme replacement therapy among developing nations is slowing down the adoption rate of enzyme replacement therapy. Also, the deficiency of technology and adequate infrastructure is expected to hamper the market growth.
  • High Cost of Enzyme Replacement Therapy
  • Stringent Reimbursement Policies

Market Segmentation

The global enzyme replacement therapy market is segmented and analyzed for demand and supply by-products into agalsidase beta, imiglucerase, velaglucerase alfa, and others, out of which, the agalsidase beta segment is anticipated to garner the largest revenue by the end of 2033, backed by increasing prevalence of chronic disease in the population across the world owing to consumption of unhealthy food items and an engaged damaging lifestyle. Between 2000 and 2030, it was predicted that the number of Americans with chronic diseases would increase by around 45 million, or roughly 36%. Furthermore, the agalsidase segment held the second-largest market share in 2023. The increased cost and growing adoption of this medication in the treatment of Fabry disease are the principal factors for this segment's substantial market share.

The global enzyme replacement therapy market is segmented and evaluated for demand and availability by disease into Gaucher disease, Fabry disease, Pompe disease, mucopolysaccharidosis, and exocrine pancreatic insufficiency [EPI], out of which, the exocrine pancreatic insufficiency segment captures the largest share in 2023 owing to the increasing incidence of this ailment amongst the general population. Furthermore, the high pervasiveness of exocrine pancreatic insufficiency among individuals suffering from chronic pancreatitis and cystic fibrosis contributes to a rise in the disease's patient group. This, in turn, contributes to this segment's supremacy over the forecast period. According to a Cleveland Clinic organization report, nearly 9 out of 10 infants with cystic fibrosis develop EPI during the first year. The remainder is in danger of developing EPI as children or adults.

Our in-depth analysis of the global enzyme replacement therapy market includes the following segments:

              By Product

  • Agalsidase Beta
  • Imiglucerase
  • Velaglucerase Alfa
  • Idursulfase
  • Galsulfase
  • Laronidase

              By Disease

  • Gaucher Disease
  • Fabry Disease
  • Pompe Disease
  • Mucopolysaccharidosis
  • Exocrine Pancreatic Insufficiency [EPI]

               By End-User

  • Hospitals
  • Infusion Centers & Home Healthcare Settings

Global Enzyme Replacement Therapy Market Regional Synopsis


The Asia Pacific enzyme replacement therapy market, amongst the market in all the other regions, is projected to hold the largest market share by the end of 2033, owing to the growing number of patient population suffering from rare disorders, rising disposable income, growth in the healthcare sector, and an increase in government support. According to the Economist Intelligence Unit, 258 million individuals in Asia and the Pacific region suffer from rare diseases.


On the other hand, in 2023, Europe held the second-largest share of the worldwide market. The increase can be credited primarily to supportive regulatory policies in a few European nations for different infrequent lysosomal ailments, including Gaucher disease, MPS, and others. As per F1000 Research Ltd., the Polish National Health Fund, for instance, implemented a national drug program in 2019, with the first remuneration in ERT for Fabry disease. Furthermore, rapid advancements in medical infrastructure and a growing preference for therapeutic approaches for rare diseases drive market expansion in this region.

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Top Featured Companies Dominating the Global Enzyme Replacement Therapy Market

    • BioMarin
      • Company Overview
      • Business Strategy
      • Key Product Offerings
      • Financial Performance
      • Key Performance Indicators
      • Risk Analysis
      • Recent Development
      • Regional Presence
      • SWOT Analysis
    • Leadiant Biosciences, Inc.
    • Pfizer Inc.
    • Sanofi Aventis
    • AbbVie Inc.
    • Takeda Pharmaceutical Company Limited
    • JCR Pharmaceutical Co., Ltd.
    • AstraZeneca plc
    • Clinigen Limited
    • Teijin Limited 


In The News


  •  Teijin Limited announced the immediate availability of Revcovi 2.4 mg for intramuscular injection [Elapegademase (Genetic Recombination)]. With Revcovi 2.4 mg, Japan's first medication for Adenosine Deaminase (ADA) Deficiency, individuals with ADA Deficiency are expected to have improved ADA activity and immune system.

  • Clinigen Limited, a global pharmaceutical Products, and Services company, announced that Clinigen K.K., a fully owned subsidiary of Clinigen located in Tokyo, has gained manufacturing and marketing authorization for Hunterase (Idursulfase-beta) ICV 15mg as part of its strategic relationship with GC Pharma. The clearance of Hunterase ICV in Japan marks its first appearance in any nation globally.



Global Economic Impact

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Despite Inflation & Fearing Recession, Businesses Across the Globe Expected to Do Better in 2023:

In 2023, market players might incur losses due to huge gap in currency translation followed by contracting revenues, shrinking profit margins & cost pressure on logistics and supply chain.
Controlling Inflation has become the first priority for global economies from last quarter of 2022 and to be followed in 2023. With skewed economic situations, rise in interest rate by governments to control spending and inflation, spiked oil and gas prices, high inflation, geo-political issues including U.S. & China trade war, Russia-Ukraine conflict to intensify the global economic issues.
The interest rates in the U.S. may be less sensitive in 2023 as compared to 2022; sigh of relief for businesses. Positive business sentiments, healthy business balance sheets, growth in construction spending (private construction value in 2022 stood at $1,429.2 billion, 11.7 percent (±1.0 percent) above the $1,279.5 billion spent in 2021, Residential construction in 2022 was $899.1 billion, up by 13.3 percent (±2.1 percent) from $793.7 billion in 2021, non-residential construction touched $530.1 billion, 9.1 percent (±1.0 percent) above the $485.8 billion in 2021.) showcases minimal impact of recession in the country.
Similarly, spiked spending in the European and major Asia economics including, India, China & Japan to showcase less impact on the global demand.

Author Credits:  Radhika Gupta, Shivam Bhutani

Key Questions Answered in the Report

1) What are the major factors driving the growth of the enzyme replacement therapy market?

Ans: The major factors driving the growth of the market are rise in prevalence of rare disease, growing government spending on healthcare infrastructure, growing cases of Pompe disease, and others.

2) What would be the CAGR of enzyme replacement therapy market over the forecast period?

Ans: The market is anticipated to attain a CAGR of ~7% over the forecast period, i.e., 2023 – 2033.

3) What are the challenges affecting the enzyme replacement therapy market growth?

Ans: Lack of awareness, high cost of enzyme replacement therapy, and stringent reimbursement policies are some of the major factors expected to hinder the growth of the market.

4) Which region will provide more business opportunities for growth of enzyme replacement therapy market in future?

Ans: The market in Asia Pacific is projected to hold the largest market share by the end of 2033 and provide more business opportunities in the future.

5) Who are the major players dominating the enzyme replacement therapy market?

Ans: The major players in the market are BioMarin, Leadiant Biosciences, Inc., Pfizer Inc., Sanofi, and more.

6) How are the company profiles selected?

Ans: The company profiles are selected based on the revenues generated from the product segment, geographical presence of the company which determine the revenue generating capacity as well as the new products being launched into the market by the company.

7) What are the segments in the enzyme replacement therapy market?

Ans: The market is segmented by product, disease, end user, and by region.

8) Which segment captures the largest market size in product in the enzyme replacement therapy market?

Ans: The agalsidase beta segment is anticipated to garner the largest market size by the end of 2033 and display significant growth opportunities.

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